The Pharmacology of Gene and Cell Therapy

von Conlon, Thomas J., Mavilio, Fulvio
Veröffentlicht in Mol Ther Methods Clin Dev (2018)

Rapid, widespread transduction of the murine myocardium using self-complementary Adeno-associated virus

von Andino, Lourdes M, Conlon, Thomas J, Porvasnik, Stacy L, Boye, Sanford L, Hauswirth, William W, Lewin, Alfred S
Veröffentlicht 2007

Apparently Nonspecific Enzyme Elevations After Portal Vein Delivery of Recombinant Adeno-Associated Virus Serotype 2 Vector in Hepatitis C Virus-Infected Chimpanzees

von Flotte, Terence R., Goetzmann, Jason, Caridi, James, Paolillo, Joseph, Conlon, Thomas J., Potter, Mark, Mueller, Christian, Byrne, Barry J.
Veröffentlicht 2008

A Preclinical Study in Rhesus Macaques for Cystic Fibrosis to Assess Gene Transfer and Transduction by AAV1 and AAV5 with a Dual-Luciferase Reporter System

von Guggino, William B., Benson, Janet, Seagrave, JeanClare, Yan, Ziying, Engelhardt, John, Gao, Guangping, Conlon, Thomas J., Cebotaru, Liudmila
Veröffentlicht in Hum Gene Ther Clin Dev (2017)

Functional Consequences of PDK4 Deficiency in Doberman Pinscher Fibroblasts

von Bolfer, Luiz, Estrada, Amara H., Larkin, Chelsea, Conlon, Thomas J., Lourenco, Francisco, Taggart, Kathryn, Suzuki-Hatano, Silveli, Pacak, Christina A.
Veröffentlicht in Sci Rep (2020)

Regression of Schwannomas Induced by Adeno-Associated Virus-Mediated Delivery of Caspase-1

von Prabhakar, Shilpa, Taherian, Mehran, Gianni, Davide, Conlon, Thomas J., Fulci, Giulia, Brockmann, Jillian, Stemmer-Rachamimov, Anat, Sena-Esteves, Miguel, Breakefield, Xandra O., Brenner, Gary J.
Veröffentlicht 2013

Preclinical Toxicology and Biodistribution Studies of Recombinant Adeno-Associated Virus 1 Human Acid α-Glucosidase

von Conlon, Thomas J., Erger, Kirsten, Porvasnik, Stacy, Cossette, Travis, Roberts, Cheryl, Combee, Lynn, Islam, Saleem, Kelley, Jeffry, Cloutier, Denise, Clément, Nathalie, Abernathy, Corinne R., Byrne, Barry J.
Veröffentlicht 2013

Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning

von Corti, Manuela, Cleaver, Brian, Clément, Nathalie, Conlon, Thomas J., Faris, Kaitlyn J., Wang, Gensheng, Benson, Janet, Tarantal, Alice F., Fuller, Davis, Herzog, Roland W., Byrne, Barry J.
Veröffentlicht in Hum Gene Ther Clin Dev (2015)

Retrograde Gene Delivery to Hypoglossal Motoneurons Using Adeno-Associated Virus Serotype 9

von ElMallah, Mai K., Falk, Darin J., Lane, Michael A., Conlon, Thomas J., Lee, Kun-Ze, Shafi, Nadeem I., Reier, Paul J., Byrne, Barry J., Fuller, David D.
Veröffentlicht 2012

Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa

von Deng, Wen-Tao, Dyka, Frank M., Dinculescu, Astra, Li, Jie, Zhu, Ping, Chiodo, Vince A., Boye, Sanford L., Conlon, Thomas J., Erger, Kirsten, Cossette, Travis, Hauswirth, William W.
Veröffentlicht in Hum Gene Ther (2015)

Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors

von Conlon, Thomas J., Mah, Cathryn S., Pacak, Christina A., Rucker Henninger, Mary B., Erger, Kirsten E., Jorgensen, Marda L., Lee, C. Chang I., Tarantal, Alice F., Byrne, Barry J.
Veröffentlicht in Hum Gene Ther Clin Dev (2016)

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

von Hauswirth, William W., Aleman, Tomas S., Kaushal, Shalesh, Cideciyan, Artur V., Schwartz, Sharon B., Wang, Lili, Conlon, Thomas J., Boye, Sanford L., Flotte, Terence R., Byrne, Barry J., Jacobson, Samuel G.
Veröffentlicht 2008

Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease

von Corti, Manuela, Liberati, Cristina, Smith, Barbara K., Lawson, Lee Ann, Tuna, Ibrahim S., Conlon, Thomas J., Coleman, Kirsten E., Islam, Saleem, Herzog, Roland W., Fuller, David D., Collins, Shelley W., Byrne, Barry J.
Veröffentlicht in Hum Gene Ther Clin Dev (2017)

Vision 1 Year after Gene Therapy for Leber’s Congenital Amaurosis

von Cideciyan, Artur V., Hauswirth, William W., Aleman, Tomas S., Kaushal, Shalesh, Schwartz, Sharon B., Boye, Sanford L., Windsor, Elizabeth A.M., Conlon, Thomas J., Sumaroka, Alexander, Roman, Alejandro J., Byrne, Barry J., Jacobson, Samuel G.
Veröffentlicht 2009

Phase I/II Trial of Adeno-Associated Virus–Mediated Alpha-Glucosidase Gene Therapy to the Diaphragm for Chronic Respiratory Failure in Pompe Disease: Initial Safety and Ventilatory...

von Smith, Barbara K., Collins, Shelley W., Conlon, Thomas J., Mah, Cathryn S., Lawson, Lee Ann, Martin, Anatole D., Fuller, David D., Cleaver, Brian D., Clément, Nathalie, Phillips, Dawn, Islam, Saleem, Dobjia, Nicole, Byrne, Barry J.
Veröffentlicht 2013

LHON Gene Therapy Vector Prevents Visual Loss and Optic Neuropathy Induced by G11778A Mutant Mitochondrial DNA: Biodistribution and Toxicology Profile

von Koilkonda, Rajeshwari, Yu, Hong, Talla, Venu, Porciatti, Vittorio, Feuer, William J., Hauswirth, William W., Chiodo, Vince, Erger, Kirsten E., Boye, Sanford L., Lewin, Alfred S., Conlon, Thomas J., Renner, Lauren, Neuringer, Martha, Detrisac, Carol, Guy, John
Veröffentlicht in Invest Ophthalmol Vis Sci (2014)

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

von Brantly, Mark L., Chulay, Jeffrey D., Wang, Lili, Mueller, Christian, Humphries, Margaret, Spencer, L. Terry, Rouhani, Farshid, Conlon, Thomas J., Calcedo, Roberto, Betts, Michael R., Spencer, Carolyn, Byrne, Barry J., Wilson, James M., Flotte, Terence R.
Veröffentlicht 2009

Glycogen Storage Disease Type Ia in Canines: A Model for Human Metabolic and Genetic Liver Disease

von Specht, Andrew, Fiske, Laurie, Erger, Kirsten, Cossette, Travis, Verstegen, John, Campbell-Thompson, Martha, Struck, Maggie B., Lee, Young Mok, Chou, Janice Y., Byrne, Barry J., Correia, Catherine E., Mah, Cathryn S., Weinstein, David A., Conlon, Thomas J.
Veröffentlicht 2011

Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year

von Cideciyan, Artur V., Hauswirth, William W., Aleman, Tomas S., Kaushal, Shalesh, Schwartz, Sharon B., Boye, Sanford L., Windsor, Elizabeth A.M., Conlon, Thomas J., Sumaroka, Alexander, Pang, Ji-jing, Roman, Alejandro J., Byrne, Barry J., Jacobson, Samuel G.
Veröffentlicht 2009

An acidic oligopeptide displayed on AAV2 improves axial muscle tropism after systemic delivery

von Lee, Ni-Chung, Falk, Darin J, Byrne, Barry J, Conlon, Thomas J, Clement, Nathalie, Porvasnik, Stacy, Jorgensen, Marda L, Potter, Mark, Erger, Kirsten E, Watson, Rachael, Ghivizzani, Steven C, Chiu, Hung-Chuan, Chien, Yin-Hsiu, Hwu, Wuh-Liang
Veröffentlicht 2012